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Management of children with congenital nephrotic syndrome: challenging treatment paradigms

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dc.contributor.authorALPAY, HARİKA
dc.contributor.authorsDufek, Stephanie; Holtta, Tuula; Trautmann, Agnes; Ylinen, Elisa; Alpay, Harika; Ariceta, Gema; Aufricht, Christoph; Bacchetta, Justine; Bakkaloglu, Sevcan A.; Bayazit, Aysun; Cicek, Rumeysa Yasemin; Dursun, Ismail; Duzova, Ali; Ekim, Mesiha; Iancu, Daniela; Jankauskiene, Augustina; Klaus, Guenter; Paglialonga, Fabio; Pasini, Andrea; Printza, Nikoleta; Conti, Valerie Said; Faria, Maria do Sameiro; Schmitt, Claus Peter; Stefanidis, Constantinos J.; Verrina, Enrico; Vidal, Enrico; Vondrak, Karel; Webb, Hazel; Zampetoglou, Argyroula; Bockenhauer, Detlef; Edefonti, Alberto; Shroff, Rukshana
dc.date.accessioned2022-03-14T10:04:55Z
dc.date.available2022-03-14T10:04:55Z
dc.date.issued2019-08-01
dc.description.abstractBackground. Management of children with congenital nephrotic syndrome (CNS) is challenging. Bilateral nephrectomies followed by dialysis and transplantation are practiced in most centres, but conservative treatment may also be effective. Methods. We conducted a 6-year review across members of the European Society for Paediatric Nephrology Dialysis Working Group to compare management strategies and their outcomes in children with CNS. Results. Eighty children (50% male) across 17 tertiary nephrology units in Europe were included (mutations in NPHS1, n = 55; NPHS2, n = 1; WT1, n = 9; others, n = 15). Excluding patients with mutations in WT1, antiproteinuric treatment was given in 42 (59%) with an increase in S-albumin in 70% by median 6 (interquartile range: 3-8) g/L (P< 0.001). Following unilateral nephrectomy, S-albumin increased by 4 (1-8) g/L (P = 0.03) with a reduction in albumin infusion dose by 5 (2-9) g/kg/week (P = 0.02). Median age at bilateral nephrectomies (n = 29) was 9 (7-16) months. Outcomes were compared between two groups of NPHS1 patients: those who underwent bilateral nephrectomies (n = 25) versus those on conservative management (n = 17). The number of septic or thrombotic episodes and growth were comparable between the groups. The response to antiproteinuric treatment, as well as renal and patient survival, was independent of NPHS1 mutation type. At final follow-up (median age 34months) 20 (80%) children in the nephrectomy group were transplanted and 1 died. In the conservative group, 9 (53%) remained without dialysis, 4 (24%; P< 0.001) were transplanted and 2 died. Conclusion. An individualized, stepwise approach with prolonged conservative management may be a reasonable alternative to early bilateral nephrectomies and dialysis in children with CNS and NPHS1 mutations. Further prospective studies are needed to define indications for unilateral nephrectomy.
dc.identifier.doi10.1093/ndt/gfy165
dc.identifier.eissn1460-2385
dc.identifier.issn0931-0509
dc.identifier.pubmed30215773
dc.identifier.urihttps://hdl.handle.net/11424/244018
dc.identifier.wosWOS:000493307500019
dc.language.isoeng
dc.publisherOXFORD UNIV PRESS
dc.relation.ispartofNEPHROLOGY DIALYSIS TRANSPLANTATION
dc.rightsinfo:eu-repo/semantics/openAccess
dc.subjectbilateral nephrectomies
dc.subjectcongenital nephrotic syndrome
dc.subjectgenotype-phenotype correlation
dc.subjectmanagement approach
dc.subjectNPHS1
dc.subjectDIFFUSE MESANGIAL SCLEROSIS
dc.subjectSTEROID-RESISTANT
dc.subjectGENOTYPE/PHENOTYPE CORRELATIONS
dc.subjectGLOMERULAR PROTEIN
dc.subject1ST YEAR
dc.subjectMUTATIONS
dc.subjectNPHS1
dc.subjectNEPHRIN
dc.subjectSURVIVAL
dc.subjectLIFE
dc.titleManagement of children with congenital nephrotic syndrome: challenging treatment paradigms
dc.typearticle
dspace.entity.typePublication
local.avesis.id7526a9e5-0d38-4d6c-8b03-d32752d642d5
local.import.packageSS16
local.indexed.atWOS
local.indexed.atSCOPUS
local.indexed.atPUBMED
local.journal.numberofpages9
local.journal.quartileQ1
oaire.citation.endPage1377
oaire.citation.issue8
oaire.citation.startPage1369
oaire.citation.titleNEPHROLOGY DIALYSIS TRANSPLANTATION
oaire.citation.volume34
relation.isAuthorOfPublication102b331c-4c62-4795-880d-234f6b72fa6a
relation.isAuthorOfPublication.latestForDiscovery102b331c-4c62-4795-880d-234f6b72fa6a

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